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Baby with rare disease given world-first personal CRISPR gene therapy

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A Baby Received a Custom Crispr Treatment in Record Time

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A Baby Receives the First Customized CRISPR Treatment

Infant becomes world's first patient to undergo personalized gene-editing treatment

A Pennsylvania baby is the first to successfully receive personalized gene therapy

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Baby Is Healed With World’s First Personalized Gene-Editing Treatment

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Baby suffering from rare disease saved with cutting-edge gene editing treatment customized to his DNA

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Gene editing helped a desperately ill baby thrive. Scientists say it could someday treat millions

Metaculus

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Gene Editing Therapy Made Just 6 Months Helps Baby Life Threatening Diseas

Jack Tseng

Eriona Hysolli

Colossal Biosciences Inc.

Beth Shapiro

Joseph Frederickson

Ben Lamm

Brian Klaas

George Church

Ben Mezrich

Mary Beth Griggs

Jeremy Austin

"[N]o one is actually going to bring mammoths back."

"Is this [the restoration of woolly mammoths] going to happen anytime soon? The answer is absolutely not."

"Mammoth de-extinction is more about media attention for the scientists and less about doing serious science."

"It feels to me that a mammoth is a long way in the future."

"I think it's [bringing back woolly mammoth] definitely within the technology."

"In 2028, if all goes according to plan, a six-foot-five narcoleptic scientist with a bushy white beard will resurrect the first living woolly mammoth in 4,000 years."

"I think the first engineered elephant will be the major milestone and that may be consistent with Ben's (Lamm) prediction of six years from 2021."

"That timeline [2028 for the birth of the first mammoth calves] hasn't changed."

"We have set a date of late 2028 for our first mammoth and we are on track."

Will the Woolly Mammoth Be Resurrected by 2028?

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KJ Muldoon of Clifton Heights, Pennsylvania, diagnosed with a rare genetic disorder called severe CPS1 deficiency, which affects one in a million infants, at two days old, has shown significant improvement after receiving a personalized CRISPR gene editing treatment at Children's Hospital of Philadelphia.

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KJ Muldoon of Clifton Heights, Pennsylvania, diagnosed with a rare genetic disorder called severe CPS1 deficiency, which affects one in a million infants, at two days old, has shown significant improvement after receiving a personalized CRISPR gene editing treatment at Children's Hospital of Philadelphia.

The medical team, led by Dr. Kiran Musunuru and Dr. Rebecca Ahrens-Nicklas, developed a customized base editing therapy within six months to correct KJ's specific genetic mutation that prevents his body from properly processing ammonia, which can be toxic to the brain.

The medical team, led by Dr. Kiran Musunuru and Dr. Rebecca Ahrens-Nicklas, developed a customized base editing therapy within six months to correct KJ's specific genetic mutation that prevents his body from properly processing ammonia, which can be toxic to the brain.

The experimental treatment, delivered through lipid nanoparticles to KJ's liver cells, involved three doses administered between February and April. He is the first to receive a personalized CRISPR therapy designed for a single individual.

Nature

The experimental treatment, delivered through lipid nanoparticles to KJ's liver cells, involved three doses administered between February and April. He is the first to receive a personalized CRISPR therapy designed for a single individual.

Following the treatment, KJ, now nearly ten months old, has reportedly shown positive responses, including improved protein tolerance, better recovery from common childhood illnesses like rhinovirus, and reduced medication needs.

Chop

Pennmedicine

Following the treatment, KJ, now nearly ten months old, has reportedly shown positive responses, including improved protein tolerance, better recovery from common childhood illnesses like rhinovirus, and reduced medication needs.

The cost of developing this custom treatment was comparable to the cost of a liver transplant procedure, which typically exceeds $800,000. The team's work was funded in part by the National Institutes of Health.

National Institutes of Health (NIH)

The cost of developing this custom treatment was comparable to the cost of a liver transplant procedure, which typically exceeds $800,000. The team's work was funded in part by the National Institutes of Health.

KJ's case is detailed in a study published by The New England Journal of Medicine on Thursday and was presented at the American Society of Gene &amp; Cell Therapy Annual Meeting in New Orleans.

KJ's case is detailed in a study published by The New England Journal of Medicine on Thursday and was presented at the American Society of Gene & Cell Therapy Annual Meeting in New Orleans.

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The medical team, led by Dr. Kiran Musunuru and Dr. Rebecca Ahrens-Nicklas, developed a customized base editing therapy within six months to correct KJ's specific genetic mutation that prevents his body from properly processing ammonia, which can be toxic to the brain.</span

NBC #5478

The experimental treatment, delivered through lipid nanoparticles to KJ's liver cells, involved three doses administered between February and April 2025, making him the first patient to receive a personalized CRISPR therapy designed for a single individual.</span

LiveNOW from FOX #

New York Post #5852

The experimental treatment, delivered through lipid nanoparticles to KJ's liver cells, involved three doses administered between February and April 2025, making him the first patient to receive a personalized CRISPR therapy designed for a single individual.

Following the treatment, KJ has shown positive responses including improved protein tolerance, better recovery from common illnesses, and reduced medication needs, though researchers emphasize it's too early to determine the full extent of the therapy's effectiveness.</span

Following the treatment, KJ has shown positive responses including improved protein tolerance, better recovery from common illnesses, and reduced medication needs, though researchers emphasize it's too early to determine the full extent of the therapy's effectiveness.

The cost of developing this custom treatment was comparable to a liver transplant procedure, which typically exceeds $800,000, suggesting potential economic viability for future personalized genetic treatments.</span

Breitbart #

The cost of developing this custom treatment was comparable to a liver transplant procedure, which typically exceeds $800,000, suggesting potential economic viability for future personalized genetic treatments.

The breakthrough represents a significant advancement in treating rare genetic disorders, potentially opening doors for millions of patients with rare conditions who have limited treatment options.</span

Science #

The breakthrough represents a significant advancement in treating rare genetic disorders, potentially opening doors for millions of patients with rare conditions who have limited treatment options.

The experimental nature of this treatment raises significant concerns about long-term safety and efficacy. Without comprehensive long-term studies, the risks of unintended genetic changes remain unknown, and the high cost of personalized treatments could create accessibility barriers for many patients who need them.

The experimental nature of this treatment raises significant concerns about long-term safety and efficacy. Without comprehensive long-term studies, the risks of unintended genetic changes remain unknown, and the high cost of personalized treatments could create accessibility barriers for many patients who need them.

Nature #

Worlds first personalized CRISPR therapy given to baby with genetic disease

Baby Makes History with Custom CRISPR Gene Therapy Success

Health

Science & technology

World

This groundbreaking achievement highlights the remarkable potential of personalized gene editing to revolutionize medicine. The swift development time and successful implementation prove that custom genetic treatments can be both feasible and practical, potentially offering hope to millions affected by rare genetic disorders who currently have no viable treatment options.

This groundbreaking achievement highlights the remarkable potential of personalized gene editing to revolutionize medicine. The swift development time and successful implementation prove that custom genetic treatments can be both feasible and practical, potentially offering hope to millions affected by rare genetic disorders who currently have no viable treatment options.

World's First Patient Treated with Personalized CRISPR Gene Editing Therapy at Childrens Hospital of Philadelphia

There's a 50% chance that at least 40 gene-edited babies will have been born worldwide by the end of 2029, according to the Metaculus prediction community.

There's a 50% chance that at least 40 gene-edited babies will have been born worldwide by the end of 2029, according to the Metaculus prediction community.

Muldoon Family via The New York Times

Custom CRISPR Gene Therapy Saves Baby with Rare Disease

Muldoon Family via The New York Times

Custom CRISPR Gene Therapy Saves Baby with Rare Disease

Custom CRISPR Gene Therapy Saves Baby with Rare Disease

Baby Makes History with Custom CRISPR Gene Therapy SuccessSaves Baby with Rare Disease

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Custom CRISPR Gene Therapy Saves Baby with Rare Disease

Baby Makes History with Custom CRISPR Gene Therapy SuccessSaves Baby with Rare Disease

The Spin

Metaculus Prediction

The Controversies

Griggs

Frederickson

Austin

Shapiro

Tseng

Klaas

Church

Hysolli

Lamm

Articles on this story

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